EU/3/12/1064

Orphan designation

On 8 November 2012, orphan designation (EU/3/12/1064) was granted by the European Commission to Takeda Global Research and Development Centre Ltd, United Kingdom, for alisertib for the treatment of ovarian cancer.

In November 2013, Takeda Global Research and Development Centre (Europe) Ltd changed name to Takeda Development Centre Europe Ltd.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

Ovarian cancer is cancer of the ovaries (two organs in the female reproductive system that produce eggs). Most ovarian cancers occur in women over the age of 50 years. Due to the absence of symptoms in the early stages of the disease, the majority of patients are diagnosed when the cancer has spread to other parts of the body.

Ovarian cancer is a life-threatening disease that is associated with poor long-term survival.

At the time of designation, ovarian cancer affected not more than 3 in 10,000 people in the European Union (EU). This was equivalent to a total of not more than 153,000 people* and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 27), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 509,000,000 (Eurostat 2012).

At the time of designation, several medicines were authorised in the EU for the treatment of ovarian cancer. The choice of treatment depended mainly on how advanced the disease was. Treatments included surgery and chemotherapy (medicines to treat cancer), particularly with platinum-based medicines.

The sponsor has provided sufficient information to show that alisertib might be of significant benefit for patients. Early studies showed improved anticancer activity when used in combination with currently authorised medicines, and a favourable survival rate has been seen in a small group of patients with ovarian cancer that was resistant or stopped responding to treatment with platinum-based medicines.

This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

The effects of alisertib have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with alisertib in patients with ovarian cancer were ongoing.

At the time of submission, alisertib was not authorised anywhere in the EU for ovarian cancer. Orphan designation of alisertib had been granted in the United States of America for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 5 October 2012 recommending the granting of this designation.

the existence of alternative methods of diagnosis, prevention or treatment;

either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.